Cell and gene therapy are forms of medical treatment that have been at the forefront of research in the last few decades. A growing field of research, cell-based therapies involve modification of a patient’s own cells or cells from a donor to fight diseases or alleviate medical conditions to help patients continue to live their full and productive lives. The technologies used to make these therapies possible have advanced tremendously over the years.
Cell and Gene Therapy
Cell and gene therapies are overlapping forms of medical treatment. While both have the same end goal, to treat and potentially cure diseases, they have slightly differing approaches. Cell therapy is the process of replacing dysfunctional or damaged cells with new, healthy ones. These cells, which can be autologous (patient-derived) or allogeneic (donor-derived), are transferred to the patient after various steps in the manufacturing process.Â
Gene therapy, on the other hand, is a process that modifies living cells for therapeutic use, by transferring genetic material to the appropriate cells to correct the DNA code or to introduce a new gene or genes that modify the cells in order to intensify the immune system’s natural response to a disease such as cancer.
A Brief History of Cell and Gene Therapy
While the field of cell therapy represents the most recent phase of the biotechnology revolution in medicine, and more specifically cancer-fighting CAR T-cell therapy, some forms of this regenerative medicine treatment have been in practice for decades. Bone marrow transplants, for example, is a stem cell therapy treatment that has been widely known about and practiced for years. While stem cells can be derived from many sources, such as umbilical cord blood and mobilized peripheral blood, bone marrow (the spongy substances that lives within our bones) derived stem cell therapy is most commonly used today and has been for more than 50 years.Â
The first transplantation of human bone marrow occurred in 1939, which was donor-derived. By 1957, the first allogeneic hematopoietic stem cell transplantation (HSCT), occurred at the Fred Hutchinson Cancer Research Center. This procedure was pioneered by E. Donnall Thomas and his team and has led to protocols used today.Â
The scientific process behind gene therapy has been used for many years in crop development. Farmers and geneticists worked together using cross pollination and genetic engineering techniques to create stronger, more resilient crops. The first gene therapy treatment given to a human patient was in 1990, and today several gene therapies have been given FDA approval, including:
- Kymriah – approved for the treatment of children and young adults with B-cell acute lymphoblastic leukemia (ALL)
- Yescarta – approved for the treatment of adults with diffuse large B-cell lymphoma (DLBCL)
- Tecartus – approved for the treatment of advanced mantle cell lymphoma (MCL)
- Breyanzi – approved in February 2021 for the treatment of adult patients with relapsed or refractory large B-cell lymphomas
Types of Cell and Gene Therapy
There are different types of cell and gene therapies that address different diseases. In fact, there are over 1,000 therapies in clinical trials, meaning that the possibility for new therapies in the future is immense. The most common type of gene therapies are:Â
- Gene addition – inserting a new copy of a gene into targeted cells to increase protein production.
- Gene correction – modifying a part of a gene to replace damaged or dysfunctional portions.
- Gene silencing – blocking the production of a specific protein.Â
- Reprogramming – introducing new cells of a specific type to change the characteristics of targeted cells.Â
- Cell elimination – destroying malignant tumor cellsÂ
The most common types of cell therapy are:
- Blood transfusions – the transfusion of red blood cells, white blood cells and platelets from a donor.
- Hematopoietic stem cell transplantation (HSCT) – more commonly known as bone marrow transplantation, this involves the administration of healthy hematopoietic stem cells to replace damaged or dysfunctional ones.
- Immune cell therapy – the use of cells in a person’s immune system to fight diseases.
Technologies Used for Cell and Gene Therapy
The cell and gene therapy industry has been revolutionized by the use of technology. In order to retrieve cells, whether they are from the patient or from a donor, the process begins with extraction, followed by cell separation (to isolate the cells that will be used for treatment), cell storage/cryopreservation (freezing cells for use in the future) and transfusion into the recipient.
Automated Technology for Cellular Processing
For cell therapies, cells can be extracted through blood or bone marrow. Bone marrow aspirate concentrate (BMAC) is a procedure that collects bone marrow from a person’s body and concentrates it to create the optimal level of stem cells. Using an automated, closed system ensures aseptic bone marrow separation and concentration, providing physicians with the highest quality final product. The PXP platforms developed by ThermoGenesis offer efficient processing and separation of cells to obtain the highest concentration of viable cells while reducing the volume of contamination from red blood cells (RBCs).
Technology for Automated Cryopreservation and Cell Storage
One of the most important aspects of regenerative medicine is the ability to cryopreserve (freeze) cells for current and future use. Many cell therapies require multiple doses and/or the use of donor cells and thus cryopreservation plays a vital role in cell therapy. To be most effective, this process should be carried out efficiently, from collection and freezing to thawing, to ensure that cells remain viable for their intended use.Â
The BioArchive® System is an excellent option for cell freezing and storage. The system provides constant temperature control through closed-system sample handling by combining integrated control rate freezing, robotic storage and retrieval. This fully-automated system boasts an up to 94 percent post-thaw cell viability with minimal exposure to transient warming events (TWE). It also provides accurate and individualized documentation for freeze and sample tracking, history and inventory in compliance with Good Manufacturing Practices (GMP) cell manufacturing requirements.
ThermoGenesis and Technologies for Cell and Gene Therapy
The PXP® and BioArchive® Systems are innovative technological breakthroughs which have been developed with the needs of laboratories and physicians in mind, providing the resources they need to ensure the highest-quality final products for their patients.Â
ThermoGenesis Holdings, Inc. is a pioneer and market leader in the development and commercialization of automated cell processing technologies for the cell and gene therapy fields. We market a full suite of solutions for automated clinical biobanking, point-of-care applications and large-scale cell processing and manufacturing with a special emphasis on the emerging CAR-T immunotherapy market. We are committed to making the world a healthier place by creating innovative solutions for those in need.Â
For more information on our entire suite of automated solutions, please contact our sales team.